Sebelipase Alfa BLA Submitted for LAL Deficiency

Synageva BioPharma announced the completion of a rolling submission of the Biologics License Application (BLA) to the FDA for sebelipase alfa as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency). The company also included a request for Priority Review in the BLA submission.

The BLA submission was based on data from the Phase 3 global, randomized, double-blind, placebo controlled trial of sebelipase alfa in children and adults with LAL Deficiency and the Phase 2/3 trial of sebelipase alfa in infants with LAL Deficiency. The Phase 3 trial met the primary endpoint of ALT normalization and demonstrated significant improvements in multiple disease-related parameters of dyslipidemia and liver injury compared with placebo. In the Phase 2/3 trial, six infants met the primary endpoint of survival at 12 months of age and five infants continue on treatment with sebelipase alfa.